FDA Approves drug for rare disease

The US Food and Drugs Administration approved a new orphan drug on February 27, two days before the first European Rare Diseases Day.

The US Food and Drugs Administration (FDA) approved a new orphan drug on February 27, two days before the first European Rare Diseases Day.

Arcalyst, which is manufactured by Regeneron Pharmaceuticals, was approved for the long-term treatment of Cryopyrin-associated periodic syndromes (CAPS) disorders such as familial cold auto-inflammatory syndrome (FCAS) and muckle-wells syndrome (MWS).

Dr Curt Rosebraugh, acting director of the FDA's office of drug evaluation, said: "Arcalyst offers new promise for this small patient population suffering disorders associated with Cryopyrin-associated periodic syndromes."

"The Orphan Drug Act - now in its 25th year - has been tremendously successful in delivering effective treatments to patients with extremely rare, but serious, diseases."

A public hearing held on March 4 at the European Parliament in Brussels saw a presentation of feedback pertaining to Rare Diseases Day compiled by the participating countries.

Rare diseases were also the focus of a European-wide campaign, which saw healthcare professionals, activists, and patients take part in a specially designated day of events on February 29.

Rare Disease Day was organised by the European Organisation for Rare Diseases' (EURODIS) Council of National Alliances, which includes 15 European member states.

The day saw people promoting awareness of rare diseases using various means to lobby decision makers for greater and more committed action to help.

EURODIS said the day was needed to raise "more funds for research and care, and more research efforts directed towards rare diseases."